Another opinion

Hi, I wanted to add a few thoughts to the excellent advice you have gotten. As you realize, there are many nurses and health care workers on this site since these diseases can affect anyone. I have the fortune to help children with a suppressed immune system in a clinic that gives a lot of IV IgG for immunodeficiency diseases as well as autoimmune diseases, so I have seen it given quite a lot in my many years working there and have seen a lot of discussion about its use and how it works. I think the most helpful thing to say is that there are two goals in autoimmune disease–to flood the system with immunoglobulin so that binding of the bad antibody is prevented and to suppress production of the bad antibody. Each person has a different amount of bad antibody (“titer”) and each antibody has a different stickiness (“affinity”), so the amount of competing (good) antibody needed is different in each person, but one has to get above this amount at the peak in order for the immunoglobulin to stop the damage to the myelin. Each person also has a different drive to produce “bad” antibody (which also can change over time as the body tries erroneously to protect itself–not realizing that it is attacking its own nervous system). At the low point (trough), one wants to have enough immunoglobulin to still “convince” the immune system that it does not need more. These two processes are different and it seems to me that this is why different people need different amounts of immunoglobulin AND different timiing to give it. Again, you need enough to flood the system so that peak allows for release/competition of good antibody for bad antibody and so that the trough amount still is enough to stop the production of more bad antibody. Immunoglobulin given to a person has risks and changes the body. It is a huge protein load and will affect total body metabolism and the viscosity (“thickness”) of the blood. Adults could not take as much immunoglobulin as Emily–there would be a risk of stroke or kidney/metabolism concerns. This is a risk in children as well and most people (any age) could not tolerate that much without serious side effects. Emily is lucky that she tolerates it well and that it works for her. I think most doctors might think of alternative treatments rather than that intensity.

As Sue pointed out, plasmapheresis removes bad antibody. It sounds like Hailey Marie’s body is determined to keep trying to remake the “bad” stuff in the confusion that happens with autoimmune diseases. IV IgG or steroids or immunosuppression helps to prevent the remaking of the bad stuff as well as repleting the body with antibodies to keep it safe. I hope that the two parts of this are clear–that you have to prevent the antibody from further damaging the body by diluting it out enough (IV IgG) or removing it (plasmapheresis) AND you also have to find a way for it not to build back up.

A third important concept I have learned about autoimmune diseases is that you want no “down time”–you want to treat aggressively enough so that you do not get symptom return because symptoms are only the tip of the iceberg and when symptoms happen, there is a lot of damage going on and you are already falling behind. Damage will accumulate and the longer it goes on the harder it is to stop and reset the system.

The fourth major point is that the immune system will reset itself, but it takes a lot of patience to do so, so you have to find what works and then keep it working for a few months and then VERY SLOWLY back down. We often tell parents that children have a very enthusiastic immune system–that tries hard to do its job. Fortunately, not many children get autoimmune diseases, but when they happen, it seems like resetting the system back to normal takes an aggressive approach, but it can probably happen better than adults–who have a stubborn immune system!!!!

All of this being said, it seems that for most people, how Kevie has been treated is the more typical way with 2 g/kg “loading” doses of immunoglobulin and the frequency of administration to prevent
“down time” or symptoms of the illness. As Dawn states, maybe start with every other week. Hailey Marie obviously needs a higher trough than she was getting. Doctors often think of peaks and troughs with certain kinds of antibiotics. If the trough is too low, you can adjust this with a higher peak dose or with a shorter interval. It is probably a bit safer to adjust interval (like done with Kevie) than peak dose, but some kids will have to have really aggressive treatment like Emily.

As to alternative treatments, if immunoglobulin is not “enough” to prevent “down time”, advocate toward pulse steroids–steroids given once a week, or once every two weeks, or other intermittent interval. this is done in a number of other autoimmune diseases, is advocated in adults for CIDP, and has a LOT less side effects than daily high doses steroids. A special problem in young children with daily moderate to high dose steroids is that they will not grow in height in addition to all the other risks of steroids. Cytoxan (cyclophosfamide) is a profound temporary immunosuppression agent and it is often used in autoimmune diseases. It is powerful and quick, but it seems that a lot of people expect it by itself to be enough, and the experience at my hospital is that it is not enough–children’s immune systems are too enthusiastic. So if cytoxan is used to stop the damage, something else to allow for resetting of the immune system should also be used. As others have pointed out, plasmapheresis can be done, but it is harder in little children like Hailey Marie and you have to have good IV access and often go far away to a specialized Children’s Hospital. Imuran and CellCept suppress production of all antibodies resulting in susceptibility to infection (immunosuppression). They are slow to act particularly in reversing the damage–so often people use a “faster acting” method in combination.

It is late and I am rambling some, but I want to go back to the four biggest points. 1. You have to stop damage with something strong enough to do that. 2. You have to prevent the formation of additional bad antibody. 3. the goal should be not “down time” so that additional damage does not occur. 4. People need to make changes very slow so that the immune system can reset itself and get back to normal.

WithHope for a cure of these diseases and all children